Title:
BBP-418 in Patients With LGMD2I
Recruitment Status:
Status Last Updated:
March 16, 2021
Clinical Phenotype(s):
Study Purpose:
This is an open label study in ambulatory and non-ambulatory subjects with LGMD2I (also known as LGMD R9). This is a study to determine the safety and tolerability of ascending dose levels of BBP-418 in those subjects.
Intervention/Treatment:
Drug: BBP-418
Phase:
Study Description:
Subjects must have been enrolled in the MLB-01-001 study to participate in this trial. Three ascending dose cohorts of 4 subjects each are planned, but a fourth cohort may be added.
Study Type:
Official Title:
An Open Label Phase 2 Study of BBP-418 in Patients With Limb Girdle Muscular Dystrophy Type 2I (MLB-01-003)
Study Start Date:
February 18, 2021
Study Completion Date:
August 5, 2021
Primary Objective(s):
- Incidence of treatment-emergent adverse events (TEAEs) that lead to dose decrease or discontinuation [ Time Frame: 90 days ]
Secondary Objective(s):
- Pharmacokinetic profile of BBP-418 by assessment of maximum concentration (Cmax) [ Time Frame: Day 1 and Day 90 ]
- Changes in pharmacodynamic parameters by assessing changes in levels of N-terminal fragment of alpha dystroglycan (α-DG) [ Time Frame: Day 1 and Day 90 ]
- Changes in pharmacodynamic parameters by assessing muscle biopsy of the tibialis anterior [ Time Frame: Day 1 and Day 90 ]
- Pharmacokinetic profile of BBP-418 by assessment of area under the curve (AUC) [ Time Frame: Day 1 and Day 90 ]
Eligibility:
Ages Eligible for Study: 12 Years to 55 Years (Child, Adult)
Sexes Eligible for Study: All
Accepts Healthy Volunteers: No
Inclusion Criteria:
- Have a body weight >30 kg
- Have a genetically confirmed diagnosis of LGMD2I and be clinically affected (defined as demonstrating clinical weakness on bedside evaluation in either a limb-girdle pattern, or in a distal extremity)
- Able to complete the 10-meter walk test in ≤ 12 seconds unaided ("moderate disease") or are with "severe disease"/non-ambulatory as defined by being unable to complete the 10-meter walk unaided in >12 seconds
- Willing to use an adequate method of contraception from time of consent through 12 weeks after last dose
- Previous enrolment in the MLB-01-001 study
Exclusion Criteria:
- Evidence of clinically significant concomitant disease, including:
- Any history of a gastrointestinal condition, including surgeries, which may affect absorption after oral administration
- Any significant concomitant medical condition, including cardiac, pulmonary, renal, hepatic or endocrine disease other than that associated with LGMD2I
- Any condition other than LGMD2I requiring therapy with prescription medicine (medication for common and mild concomitant conditions may be permitted after consultation with the PI and the Medical Monitor)
- Any other laboratory, vital sign, ECG abnormality, or clinical history or finding that, in the investigator's opinion, is likely to unfavorably alter the risk-benefit of study participation, confound study results, or interfere with study conduct or compliance
- If pregnant, planning to become pregnant and/or breastfeeding or planning to conceive children within the projected duration of the study through 12 weeks after the last dose of study treatment.
- History of drug abuse including alcoholism within 2 years prior to consenting
- Use of ribose or other sugar alcohol-containing supplement within 60 days of Day 1
- Use of a corticosteroid within 60 days of Day 1
- Presence of a platelet disorder, bleeding disorder or other contraindication to muscle biopsy
- Actively on an experimental therapy or device or was on an experimental therapy or device within 60 days prior to Day 1.
Study Site(s)/Location(s):
United States, Virginia
Virginia Commonwealth University
Richmond, Virginia, United States, 23219
Sponsors & Collaborators:
ML Bio Solutions, Inc.
Principal Investigator(s):
Principal Investigator:
Nicholas Johnson, MD
Virginia Commonwealth University
For more information, please contact the Study Coordinator:
Contact:
Email:
Phone:
ClinicalTrials.gov ID:
NCT04800874